Safety and Efficacy of RRG001 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: RRG001.
- Who it may be relevant to
- Registry conditions: Neovascular Age-related Macular Degeneration. Basic parameters: from 50 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Phase I/IIa, Dose-escalation and Dose-extension Study to Evaluate the Safety and Efficacy of Single Subretinal Injection of RRG001 in Subjects With Neovascular Age-related Macular Degeneration
Overview
A Phase I/IIa, Dose-escalation and Dose-expansion Study to Evaluate the Safety and Efficacy of Single Subretinal Injection of RRG001 in Subjects With Neovascular Age-related Macular Degeneration
Detailed description
This study adopts a multicenter, single-arm, open-label phase I/IIa seamless study design and comprises two stages, i.e., phase I 3+3 dose escalation study and a phase IIa dose expansion study. Four dose groups are preset for the phase I dose escalation study; 3-6 subjects will be enrolled for each dose group, and it is planned to enroll 12-24 subjects. The phase IIa dose expansion study will be conducted successively at the doses after safety assessments of dose 2 and/or dose 3 in phase I are completed. Twelve subjects are expected to be enrolled for each dose group, with a total of 24 subjects will be enrolled overall. The sponsor will assess whether or not to adjust the dose(s) and sample size for the exploration study based on the results in the phase I exploration study.
Interventions
- Drug RRG001
Administered via Subretinal injection. Dosage form: injection solution.
Primary outcome measures
- Safety and tolerability [Time frame: Week 52]
Secondary outcome measures (2)
- Efficacy after RRG001 injection [Time frame: Week 52]
- Efficacy after RRG001 injection [Time frame: Week 52]
Eligibility criteria
Inclusion criteria
- Subjects that are willing and able to follow study procedures.
- Patinets ≥50 years old.
- Clinically diagnosed with CNV secondary to nAMD.
- BCVA of the study eye is ≥5 letters and ≤73 letters(Snellen 20/800\~20/40), as well as that of the other eye ≥19 letters(Snellen 20/400).
- Responding to anti-VEGF
Exclusion criteria
- Presence of any other eye diseases other than nAMD in study eye that would affect the treatment or confusing the interpretation of the study results , as judged by the investigator.
- CNV or macular edema in the study eye secondary to any causes other than AMD.
- Uncontrolled glaucoma.
- Uncontrolled hypertension despite medication.
- Pregnant or lactating women or individuals with reproductive potential who are unwilling to take effective contraception during the study period.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Tianjin Medical University Eye Hospital — Tianjin
Identifiers
NCT: NCT06141460 · RRG001-101